Eight presentations highlight the differentiated profile and broad applicability of the company’s HSC gene therapy platform
Data from proof-of-concept study of OTL-203 in MPS-IH demonstrate favorable outcomes for clinical manifestations of the disease not well addressed by the current standard-of-care
Chief Technical Officer
Featured presentations include a range of interim clinical outcomes in addition to the neurological and skeletal results previously reported from the company’s proof-of-concept study of OTL-203 in the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), as well as well as a first look at pre-clinical data demonstrating the use of vectorized HSCs as a delivery vehicle for monoclonal antibodies. In addition, several abstracts from the company’s pre-clinical research programs demonstrate the therapeutic potential of HSC gene therapy to address larger indications, including a genetic sub-type of frontotemporal dementia (FTD) and Crohn’s disease, as well as chronic autoimmune disorders.
The company will also host a sponsored symposium on
Additionally,
“Together with our clinical collaborators, we’re proud of our significant presence at ESGCT which continues to underscore the transformative impact and broad applicability of our HSC gene therapy platform,” said
Details of the oral presentations are follows (all times in CEST):
- Title: Lentiviral ex-vivo HSC gene therapy as a tool to deliver therapeutic antibodies beyond the blood-brain barrier
Date/Time:Tuesday, October 24 at6:15 p.m.
Lead Author:Chiara Recchi
- Title: Interim skeletal outcome after hematopoietic stem and progenitor cell-gene therapy for Mucopolysaccharidosis type I Hurler
Date/Time:Wednesday, October 25 at3:30 p.m.
Lead Author:Maria Ester Bernardo
- Title: Development of an ex vivo hematopoietic stem cell Gene Therapy for Frontotemporal Dementia (FTD)
Date/Time:Friday, October 27 at9:45 a.m.
Lead Author:Yuri Ciervo
Details of the poster presentations are as follows (all times in CEST):
- Title: Hematopoietic Stem Cell Gene Therapy as a Novel Gene Therapy Approach for Severe Crohn’s Disease Associated with NOD2-Deficiency
Date/Time:Wednesday, October 25 at5:00 p.m.
Lead Author:Florence Enjalbert
- Title: HSC-derived CAR-Treg gene therapy: a novel approach for the treatment of chronic autoimmune disease
Date/Time:Wednesday, October 25 at5:00 p.m.
Lead Author:Greg Crawford
- Title: Interim non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy for Mucopolysaccharidosis type I Hurler
Date/Time:Wednesday, October 25 at6:15 p.m.
Lead Author:Francesca Tucci
- Title: Dissecting the mechanisms of myeloid-to-neural enzymatic cross-correction in the context of hematopoietic stem cell gene therapy for Metachromatic Leukodystrophy
Date/Time:Wednesday, October 25 at5:00 p.m.
Lead Author:Vasco Meneghini
- Title: Downstream strategies for concentration and purification of LV Vectors from suspension cell-based systems
Date/Time:Wednesday, October 25 at6:15 p.m.
Lead Author:Luca Crippa
About
At
In 2018, the company acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the
Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit www.orchard-tx.com, and follow us on X (Twitter) and LinkedIn.
Availability of Other Information About Orchard
Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (X; Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchard’s investor relations website and may include additional social media channels. The contents of Orchard’s website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.
Forward-looking Statements
This press release contains forward-looking statements, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchard’s products and product candidates. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchard’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation, the risk that products will not be successfully commercialized, and the risk that long-term adverse safety findings may be discovered. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.
Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchard’s most recent annual or quarterly report filed with the
ContactBenjamin Navon +1 857-248-9454 Benjamin.Navon@orchard-tx.com
Source:
2023 GlobeNewswire, Inc., source